UO Neurogenetica Molecolare, Fondazione IRCCS Isitituto Neurologico C. Besta, Via Celoria 11, 20100 Milano, Italy.
Curr Pharm Des. 2010;16(8):978-87. doi: 10.2174/138161210790883471.
Muscle degeneration and regeneration are two of the most evident pathological events characterizing muscular diseases and in particular muscular dystrophies. Muscular dystrophies are an heterogeneous group of hereditary diseases affecting both children and adults, and are characterized by muscle wasting and weakness. Until now at least 30 different genes have been associated with muscular dystrophies. They have been divided into several subgroups depending on the distribution of the muscle weakness. Thus, the histopathological markers of all these forms are dystrophic changes at the muscle biopsy characterized by fiber size variability, fibres necrosis, regeneration, inflammation and connective tissues deposition. As for now, no effective therapy is available for these diseases but new inside has now been expanded in regenerative therapy such as cell therapy and gene therapy. This review is focused on muscular dystrophies and new acknowledgments in regenerative therapy.
肌肉退化和再生是肌肉疾病，尤其是肌肉营养不良症的两个最明显的病理事件。肌肉营养不良症是一组影响儿童和成人的遗传性疾病，其特征是肌肉萎缩和无力。到目前为止，至少有 30 种不同的基因与肌肉营养不良症有关。它们根据肌肉无力的分布情况分为几个亚组。因此，所有这些形式的组织病理学标志物都是肌肉活检中的营养不良变化，其特征是纤维大小变异性、纤维坏死、再生、炎症和结缔组织沉积。到目前为止，这些疾病还没有有效的治疗方法，但再生疗法，如细胞疗法和基因疗法，已经有了新的进展。本文综述了肌肉营养不良症和再生疗法的新进展。
